IND Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System: Guidance for Industry and FDA Staff

Description

We, the Center for Biologics Evaluation and Research (CBER), FDA, are providing advice to you, potential sponsors (e.g., cord blood banks, registries, transplant centers, or individual physicians serving as sponsor-investigators), to assist in the submission of an Investigational New Drug Application (IND) for certain hematopoietic progenitor cells from placental/umbilical cord blood (HPC, Cord Blood)1, when such HPC, Cord Blood units are not licensed in accordance with Title 21 of the Code of Federal Regulations Part 601 (21 CFR Part 601), and when a suitable human leukocyte antigen (HLA) matched cord blood transplant is needed for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment and there is no satisfactory alternative treatment available. If unlicensed HPC, Cord Blood units are made available for clinical use, they must be distributed under an IND meeting the applicable requirements in 21 CFR Part 312.

Scope & Applicability

Regulatory Context

• 21CFR1271.60§ 1271.60 What quarantine and other requirements apply before the donor-eligibility determination is complete?

  (a) Quarantine. You must keep an HCT/P in quarantine, as defined in § 1271.3(q) , until completion of the donor-eligibility determination required by § 1271.50 . You must quarantine semen from anonymous donors until the retesting required under § 1271.85(d) is complete.Read full regulation →

• 21CFR312.8§ 312.8 Charging for investigational drugs under an IND.

  (a) General criteria for charging.Read full regulation →

• 21CFR1271.3§ 1271.3 How does FDA define important terms in this part?

  The following definitions apply only to this part:Read full regulation →

• 21CFR1271.65§ 1271.65 How do I store an HCT/P from a donor determined to be ineligible, and what uses of the HCT/P are not prohibited?

  (a) Storage. If you are the establishment that stores the HCT/P, you must store or identify HCT/Ps from donors who have been determined to be ineligible in a physically separate area clearly identified for such use, or follow other procedures, such as automated designation, that are adequate to prevRead full regulation →

• 21CFR312.40§ 312.40 General requirements for use of an investigational new drug in a clinical investigation.

  (a) An investigational new drug may be used in a clinical investigation if the following conditions are met:Read full regulation →

• 21CFR314.420§ 314.420 Drug master files.

  (a) A drug master file is a submission of information to the Food and Drug Administration by a person (the drug master file holder) who intends it to be used for one of the following purposes: To permit the holder to incorporate the information by reference when the holder submits an investigationalRead full regulation →

• 21CFR312.6§ 312.6 Labeling of an investigational new drug.

  (a) The immediate package of an investigational new drug intended for human use shall bear a label with the statement “Caution: New Drug—Limited by Federal (or United States) law to investigational use.”Read full regulation →

• 21CFR1271.55§ 1271.55 What records must accompany an HCT/P after the donor-eligibility determination is complete; and what records must I retain?

  (a) Accompanying records. Once a donor-eligibility determination has been made, the following must accompany the HCT/P at all times:Read full regulation →

• In Vivo Bioavailability-Bioequivalence Studies – Clinical

  Maria A. Carballosa, M.D.

  2025-12-23

• Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)

  Celularity, Inc

  2025-12-09

• Sponsor/Investigator

  Verdure Sciences, Inc.

  2025-11-18

• Clinical Investigator (Sponsor)

  Pamela K. Den Besten, DDS, MS

  2025-09-30

• Clinical Investigator/Sponsor

  Ralph A. DeFronzo, M.D.

  2025-09-23

• Clinical Investigator

  Shirish M. Gadgeel, M.D.

  2025-09-09

• Clinical Investigator

  Mark J. Savant, M.D

  2025-07-15

• Clinical Investigator

  Peter Michael, M.D.

  2025-07-01

• Clinical Investigator (Sponsor)

  American Behavioral Research Institute, LLC

  2025-06-10

• Bioresearch Monitoring Program

  Amy Lightner, MD

  2025-06-03

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